Branson Michael, Whitehead John
Novartis Pharma AG, Lichtstrasse 35, CH-4056, Basel, Switzerland.
Stat Med. 2003 Oct 30;22(20):3115-32. doi: 10.1002/sim.1542.
A score test is developed for binary clinical trial data, which incorporates patient non-compliance while respecting randomization. It is assumed in this paper that compliance is 'all-or-nothing', in the sense that a patient either accepts all of the treatment assigned as specified in the protocol, or none of it. Direct analytic comparisons of the adjusted test statistic for both the score test and the likelihood ratio test are made with the corresponding test statistics that adhere to the intention-to-treat principle. It is shown that no gain in power is possible over the intention-to-treat analysis, by adjusting for patient non-compliance. Sample size formulae are derived and simulation studies are used to demonstrate that the sample size approximation holds.
针对二元临床试验数据开发了一种计分检验，该检验在尊重随机化的同时纳入了患者的不依从性。本文假设依从性是“全有或全无”的，即患者要么接受方案中指定分配的全部治疗，要么全部不接受。对计分检验和似然比检验的调整后检验统计量与遵循意向性分析原则的相应检验统计量进行了直接的分析比较。结果表明，通过对患者的不依从性进行调整，在功效上不会比意向性分析有任何提高。推导了样本量公式，并通过模拟研究证明样本量近似成立。
